Attendees at the 2014 RAPS Convergence closing plenary session will hear from four respected thought leaders about the global regulatory healthcare market. The panelists will discuss the complexity of delivering healthcare products to consumers around the world in a safe and cost-effective manner while dealing with shifting regulatory paradigms, regulatory science and regulatory capacity.

Speaking at the plenary will be Martha Brumfield, PhD, from the Critical Path Institute; Mike Ward, Health Canada; Murray Lumpkin, MD, MSc, Bill & Melinda Gates Foundation: and John Lim, Singapore Ministry of Health and the Duke-National University of Singapore Graduate Medical School.

One issue likely to come up during the session is how different healthcare models deal with benefit-risk and risk mitigation strategies. Here’s what Lim and Lumpkin had to say on the topic when asked recently by RAPS.

“Risk-benefit and risk-mitigation strategies are important because the tendency in the past has been for more ‘one-size-fits-all’ approaches,” Lim said. “Apart from the socio-political considerations that make a risk-based approach important, resource constraints and legal considerations also necessitate the practical application of risk assessment to marketing authorization and regulatory decision-making and actions.

Use of Science-Based Risk Categorization Templates 

Regulatory agencies should first look at systematic, science-based risk categorization templates that exist and which are appropriate and applicable to their circumstances,” Lim said. He cited Singapore’s Health Sciences Authority as an example of one such template. Singapore “applies a practical, risk-based matrix and maps intrinsic and extrinsic risks of healthcare products against appropriate pre- and postmarketing regulatory approaches. This helps clarify broad categories of risks and then enables the agency to focus on specific issues,” he explained.

Quandary of Uncertainty in Low- and Middle-Income Countries

Specifically for low- and middle-income countries, Lumpkin said, “You have the known risks and the known benefits that you learn from the preclinical and clinical work, but then you have this tremendous amount of uncertainty both with respect to risks and with respect to benefits. The issue that really becomes paramount is what is the tolerance within your community for uncertainty.”

“When you’re dealing with serious and life-threatening illnesses, such as HIV and cancer where people feel they don’t have a lot of choices for their treatment, their tolerance for uncertainty is pretty high, and you need a regulatory system that is not ‘one-size-fits-all,’” Lumpkin said.

“The counterpart to that uncertainty and the challenge to that among lower and middle-income countries is that you can’t have it both ways,” he noted. “You can’t have this increased tolerance for uncertainty and, if things go bad, blame the regulators.” This is a “community decision” on therapeutic outcomes, Lumpkin said. “This is a community learning and saying, ‘We know this might turn out badly, but we’re willing to tolerate this uncertainty.’"

The “challenge” in low- and middle-income countries comes with products that are authorized in high-income countries using accelerated approval in the US or conditional authorization in the EU, Lumpkin continued. These products “are often bound to risk-mitigation plans that are very formal and that utilize the pharmacovigilance systems and the healthcare systems” that exist in high-income contexts. That is “sometimes very difficult in low- and middle-income countries to replicate,” Lumpkin said. “Can you really use those same mechanisms on the front end to authorize a healthcare product if you don’t have the system on the back end to do the risk mitigation, and what does that mean to access and different approaches that regulatory agencies take?”

The session is sure to raise plenty of questions from the audience, and there will be time allotted for Q&A. The closing plenary is from 10:30 am–12:00 pm in Ballroom D, Level 4, at the Neal Kocurek Memorial Austin Convention Center.